Pfizer presents data from Phase 2 study of ponsegromab in with cancer cachexia

Pfizer announced its Phase 2 study of ponsegromab, a monoclonal antibody directed against growth differentiation factor-15, met its primary endpoint of change from baseline in body weight compared to placebo in people with cancer cachexia and elevated levels of GDF-15. Cachexia is a common, life-threatening wasting condition that can significantly impact quality of life in affected patients with cancer. Results were presented as a late-breaking Proffered Paper Presentation at the European Society for Medical Oncology 2024 Congress and have simultaneously been published in The New England Journal of Medicine. The Phase 2 study included 187 participants with non-small cell lung cancer, pancreatic cancer, or colorectal cancer. Ponsegromab demonstrated significant and robust increases in body weight after 12 weeks across all doses: 2.02% in the 100 mg treatment group, 3.48% in the 200 mg group, and 5.61% in the 400 mg group, compared to placebo. Improvements across multiple domains of the cachexia phenotype were observed in the 400 mg group including in measures of appetite and cachexia symptoms, physical activity and skeletal muscle index. No clinically significant adverse trends were noted with ponsegromab administration. Treatment related adverse events occurred in 8.9% of patients taking placebo and 7.7% of patients taking ponsegromab. Based on these positive results, Pfizer is discussing late-stage development plans with regulators with the goal of starting registration-enabling studies in 2025. Ponsegromab is also being investigated in a Phase 2 study in patients with heart failure and elevated serum GDF-15 concentrations.